Genetic testing using a panel of DNA that represents a diverse array of genes that may cause cholestasis

Early diagnosis and treatment are very important
If left untreated, Bile Acid Synthesis Disorders can cause liver damage and eventually the liver may no longer work normally and a liver transplant may be necessary. It is important to diagnose and treat Bile Acid Synthesis Disorders as early as possible, since untreated patients may develop serious liver disease/liver failure.1

Early identification and initiation of therapy may lead to better outcomes.1,6

Bile acid synthesis disorders should be
diagnosed as early as possible

Talk to your doctor about treatment options
Bile Acid Synthesis Disorders cannot be treated by lifestyle changes. One of the primary bile acids normally produced in the liver is called cholic acid. Since patients with Bile Acid Synthesis Disorders do not properly produce this substance, they are given cholic acid as a replacement therapy. This can help restore normal liver function.6

FREE Genetic Screening for Cholestasis for Qualifying
Patients

Retrophin is committed to providing support to patients,
families and caregivers. Retrophin is offering patients free genetic
testing to help find potentional causes of cholestasis. Your doctor can gain
access to this program by visiting testcholestasis.com.
We encourage you to speak to your doctor about this test and whether or not you may be a candidate.

FREE Atypical Bile Acid Test for Cholestasis for
Qualifying Patients

Has your child been tested for toxic or harmful bile
acids associated with causing cholestasis?
The atypical bile acid test is not a routine test your doctor has likely run.
This simple urine test is important to consider if your child has cholestasis or
unexplained liver disease.
Retrophin is invested in the well-being of patients with bile acid synthesis
disorders and has partnered with Cincinnati Children's Hospital Medical Center
to offer a free laboratory test.
Download the Atypical Bile Acid Test form and talk to your child’s physician
about running this important test.

Limitation of Use:
The safety and effectiveness of CHOLBAM® on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects or peroxisomal disorder including Zellweger spectrum disorders have not been established.

Important Safety Information
Warnings and Precautions—Exacerbation of Liver Impairment

Monitor liver function and discontinue CHOLBAM® (cholic acid) in patients who develop worsening of liver function while on treatment.

Monitor AST, ALT, GGT, alkaline phosphatase, bilirubin, and international normalized ratio (INR) every month for the first 3 months, every 3 months for the next 9 months, every 6 months during the next 3 years and annually thereafter. Administer the lowest dose that effectively maintains liver function.

Discontinue CHOLBAM® if liver function does not improve within 3 months of starting treatment, if complete biliary obstruction develops, or if there are persistent clinical or laboratory indicators of worsening liver function or cholestasis; continue to monitor liver function and consider restarting at a lower dose when parameters return to baseline.

Adverse Reactions
In the CHOLBAM® clinical trials, diarrhea was the most common adverse reaction in approximately 2% of the patient population. All other adverse reactions are less than or equal to 1% of the patient population.

Bile Acid Resins and Aluminum-Based Antacids: Take CHOLBAM® at least 1 hour before or 4 to 6 hours (or at as great an interval as possible) after a bile acid binding resin or aluminum-based antacids.

Pregnancy
No studies in pregnant women or animal reproduction studies have been conducted with CHOLBAM®. Women who become pregnant during CHOLBAM® treatment are encouraged to call 1-844-202-6262.

Lactation
Endogenous cholic acid is present in human milk. Clinical lactation studies have not been conducted to assess the presence of CHOLBAM® in human milk, the effects of CHOLBAM® on the breastfed infant, or the effects of CHOLBAM® on milk production.

There are no animal lactation data and no data from case reports available in the published literature.

Overdosage
In the event of overdose (elevated GGT and ALT), the patient should be monitored and treated symptomatically.